Although this section is open to the public, the information within is intended for medical professionals and highlights some of the latest clinical and research activities of our staff. If you have any questions about your particular situation, you should speak to a physician in your area.
For information geared towards patients, visit our section on Understanding Your Illness.
* Weill Cornell Researchers May Have Identified Gene Responsible for Mutated Red Blood Cells: Avoiding_Spleen_Removal_for_Cooley_-_Rivella.doc
*Read about how Dr. Rivella is making advances in Beta-Thalassemia
*Read about Dr. Bussel’s study as published in The Lancet in 2008 ”Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial.”
*Read Dr. Bussel’s first authored paper featured in 2007 in the prestigious journal, New England Journal of Medicine ”Eltrombopag for the Treatment of Chronic Idiopathic Thrombocytopenic Purpura.”
*Learn more about Dr. Aledo’s work with leukemia that was published in Blood in 2007 “Improved Early Event Free Survival (EFS) in Children with Philadelphia Chromosome-Positive (Ph+) Acute Lymphoblastic Leukemia (ALL) with Intensive Imatinib in Combination with High Dose Chemotherapy: Children’s Oncology Group (COG) Study AALL0031”
*Dr. Bussel’s breakthroughs for Chronic ITP, featured in New England Journal of Medicine in 2006 “AMG 531, a Thrombopoiesis-Stimulating Protein, for Chronic ITP.”
*Dr. Hilgartner and Dr. DiMichele’s discoveries for hemophilia, in Haemophilia in 2003 ”Long-term FEIBA prophylaxis does not prevent progression of existing joint disease”
*Read Dr. DiMichele’s first authored article on liver biopsies for patients with bleeding disorders, published in Haemophilia in 2003 “Transjugular liver biopsy is safe and diagnostic for patients with congenital bleeding disorders and hepatitis C infection”
* Immune Tolerance in Hemophilia A/High Titer Inhibitors: (Donna DiMichele, M.D.) For the past three years as a co-principal investigator for this project, Dr. DiMichele has been developing the first international multicenter prospective randomized study of immune tolerance therapy in hemophilia A patients who develop high titer inhibitors (antibodies). This trial began in the summer of 2002. Although this dosing trail is clinical in scope, it will provide the opportunity to study the role of genotype immunophenotype interaction in inhibitor formation and disappearance through several collaborative sub-studies.
* Laboratory Predictors of Thrombosis and End-Organ Failure in Pediatric Patients Undergoing Stem Cell Transplant: (Donna DiMichele, MD). In this American Cancer Society-sponsored study, currently in the stage of final data collection and analysis, clinical laboratory parameters of hypercoagulability were measured longitudinally, pretransplant through engrafment. Through the correlation of several prethrombotic laboratory parameters and clinical even endpoints, the study will attempt to identify one or more clinical laboratory predictors of thrombosis and end-organ failure. Data from this study will form the basis for a prophylactic intervention study of anticoagulation.
* The Use of a Computational Model as a Predictor of Bleeding in Severe Hemophilia A: (Leonardo Brandao, MD). The purpose of this study is to investigate whether the use of this new computerized method of blood clotting analysis can predict/explain the bleeding tendencies in children with severe hemophilia A. Additionally, this model will be tested to see if it can also accurately predict bleeding tendencies in children with severe hemophilia A who develop an inhibitor, a substance or antibody made by the body against FVIII.
* The FEIBA (NovoSeven, Comparative Study, The FENOC Study): (Donna DiMichele, MD). Our HTC is the coordinating US/Canadian center for this study. The purpose of this study is to compare the effectiveness of FEIBA and NovoSeven in the treatment of joint bleeds in hemophilia A patients with inhibitors.
* Hemostasis Fellowship Program: A fourth year pediatric hematology/oncology fellowship with a clinical/clinical research focus on hemophilia and general hemostasis and thrombosis has been established at the Weill Cornell institution and is overseen by Dr. DiMichele. Our first candidate’s fellowship is under way for the 2002-2003 academic year. This fellow will be offered an intensive combined clinical, laboratory and research experience with the goal of developing the following expertise: 1) diagnosis/management of hemophilia, vWd, other congenital/acquired bleeding disorders as well as congenital/acquired thrombophilia in the pediatric adult patient: and 2) technical/interpretive aspects of the clinical coagulation lab. His clinical research will be co-mentored by the Children’s Clinical Research Center and accompanied by coursework in epidemiology, statistics and research design.
* Our Hemophilia Treatment Center is a designated International Hemophilia Training Center through the World Federation of Hemophilia. Annual fellowships are granted to physicians in other countries who have an interest in learning more about comprehensive care for patients with bleeding disorders. During a course of training from 4-8 weeks candidates are given the opportunity to participate in comprehensive clinics at several of the HTC’s in our region, even traveling as far as Puerto Rico, to get a better understanding of the services available at a comprehensive care center and the diverse population involved. Two trainees from Brazil spent two months with us in 2002. In 2003, we had two trainees from the Philippines for the same period of time.
